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tablets equivalent to 10mg amifampridine, 3 to 4 times per day. Initially, the daily amifampridine doses were to be the doses that were determined at the end of the Run-in Period from the MSK-002 study. The usual range was from 30 to 80 mg total daily dose, given in three (3) or four (4) doses, with no single dose \> 20 mg

tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

Amifampridine Phosphate

Renato Mantegazza, MD

Evaluate the long-term safety of amifampridine phosphate in patients with MuSK antibody positive and AChR antibody positive myasthenia gravis.

The Primary objective :

To characterize the long-term safety and tolerability of amifampridine phosphate in patients with MG

The Secondary Objective:

To assess the clinical efficacy of amifampridine phosphate over time in patients with MG based on change in Myasthenia Gravis Activities of Daily Living Score (MG-ADL)

This was a long-term extension study for subjects who participated in Protocol MSK-002 where the efficacy and safety of amifampridine was evaluated in subjects diagnosed with MuSK MG or AChR-MG. The optimal dose and schedule for amifampridine from the end of the Run-in Period from Study MSK-002 was initially used for each patient. The Investigator could adjust the dose of amifampridine during the course of the trial, in order to optimize neuromuscular benefit for the patient. Clinic visits for safety assessment and for evaluation of MG-ADL were made at Months 3, 6, 9, 12, 15, 21, 27, 33 and 39. Additional visits could occur at the discretion of the Investigator.

The Original protocol (12 Nov 2017) included analysis of MG-ADL change over time, however, Statistical Analysis Plan (SAP), dated 21 Feb 2023, removed the analysis of MG-ADL score change over time.

Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)

Long Term Safety Study of Amifampridine Phosphate in Patients With MuSK Antibody Positive and AChR (Acetylcholine Receptor) Antibody Positive Myasthenia Gravis

Evaluate the long-term safety and tolerability of amifampridine phosphate through the number of patients with treatment-emergent adverse events mapped to the Medical Dictionary for Regulatory Activities (MedDRA) SOCs and PTs. \[ Time Frame: over 39 months \] Descriptive statistics will be used to summarize study data.

Catalyst Pharmaceuticals, Inc.

Serious classification based on the FDA regulatory definition of a serious AE.

Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day.

Serious Adverse Event

tablets equivalent to 10mg amifampridine, 3 to 4 times per day. Initially, the daily amifampridine doses were to be the doses that were determined at the end of the Run-in Period from the MSK-002 study. The usual range was from 30 to 80 mg total daily dose, given in three (3) or four (4) doses, with no single dose \> 20 mg

Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

Age, Categorical

Age, Continuous

Sex: Female, Male

Race (NIH/OMB)

United States

Italy

Region of Enrollment

Baseline weight not recorded for 4 participants.

Weight

The study was terminated due to lack of measure appropriate data. The analysis of the MG-ADL score was removed in Statistical Analysis Plan (SAP), dated 21FEB2023.

Gary Ingenito, MD, Chief Medical Officer

Treatment Emergent Adverse Events

Number of Participants With Treatment-Emergent Adverse Events (Safety and Tolerability)

Overall Study

200 mL intravenous infusion on Day 1 and Day 8: CNP-106

CNP-106 is comprised of an antigenic AChR Peptide Pool (\~1 μg of each AChRα and AChRε peptide comprising AChR Peptide Pool Drug Substance per mg particles) dispersed within a negatively charged (-30 to -60 mV) polymer matrix of PLGA (Poly (DL-lactide-co-glycolide, 50:50 acid-end group)) particles (400-800 nm in size).

CNP-106

Placebo

Jason Penix

Lynlee Muehring

Nicole Turcotte

Shafeeq Ladha, MD

Lucia Rodriguez

Kumaraswamy Sivakumar, MD

Danielle Mendoza

Derrick Florin, MD

Archita Patel

Ali A Habib, MD

Erika Renkl

Bhaskar Roy, MD

Hector Fernandez

Sergio Jaramillo, MD

Naraly Requena

Tuan Vu, MD

Abby Davis

Mazen DiMachkie

Sarah Hilbert

Jeffrey Allen, MD

Julie Agriesti

Miriam Freimer, MD

Alison Line

Katherine Ruzhansky, MD

Amy Megerle

Aziz Shaibani, MD

Taylor Parkinson

Gordon Smith, MD, FAAN

Roy First, MD

Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106.

This is a Phase 1b/2a First-in-Human (FIH) clinical trial to assess the safety, tolerability, pharmacodynamics (PD), and efficacy of multiple ascending doses of CNP-106. The clinical study lasts 222-days (up to 42 days for Screening, 180 Study Days). Subjects ages 18-75 with generalized myasthenia gravis (MG) will be screened up to 42 days prior to enrollment into the clinical study.

Evaluate the Safety, Tolerability, Pharmacodynamics and Efficacy of CNP-106 in Subjects With Myasthenia Gravis

A Phase 1b/2a Double Blind, Placebo Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Efficacy of CNP-106 in Subjects Ages 18-75 With Generalized Myasthenia Gravis

Frequency tables will be presented by treatment group for all AEs and SAEs by System Organ Class (SOC) and Preferred Term (PT). Frequency tables will also be produced by treatment group for AEs leading to COUR Pharmaceuticals Development Company, Inc. Confidential CNP-106-5.001 Protocol; IND 28774 Page 53 of 65 discontinuation from IP and study, by severity, and by causality. No formal statistical testing will be done.

The mean change from Baseline to the endpoint within CNP-106 and Placebo treatment groups will be analyzed using ANOVA.

COUR Pharmaceutical Development Company, Inc.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization	Link
Long Term Safety Study of Amifampridine Phosphate in MuSK-MG (Muscle Specific Tyrosine Kinase Myasthenia Gravis)	NCT03579966	Myasthenia Grav... AChR Myasthenia...	Amifampridine P...	18 Years -	Catalyst Pharmaceuticals, Inc.
Evaluate the Safety, Tolerability, Pharmacodynamics and Efficacy of CNP-106 in Subjects With Myasthenia Gravis	NCT06106672	Myasthenia Grav... Generalized Mya... AChR Myasthenia... MuSK MG	CNP-106 Placebo	18 Years - 75 Years	COUR Pharmaceutical Development Company, Inc.

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